Publications by authors named "Solange Corriol-Rohou"

14 Publications

  • Page 1 of 1

Objectively Measured Physical Activity in Patients with COPD: Recommendations from an International Task Force on Physical Activity.

Chronic Obstr Pulm Dis 2021 08 25. Epub 2021 Aug 25.

Reval Rehabilitation Research Center, Biomed Biomedical Research Institute, Hasselt University, Diepenbeek, Belgium.

Physical activity (PA) is of key importance for health among healthy persons and individuals with COPD. PA has multiple dimensions that can be assessed and quantified objectively using activity monitors. Moreover, as shown in the published literature, variable methodologies have been used to date to quantify PA among individuals with COPD, precluding clear comparisons of outcomes across studies. The present paper aims to provide a summary of the available literature for the rationale behind using objectively measured PA and proposes a standardized methodology for assessment, including standard operating procedures for future research. The present paper therefore describes the concept of PA, reports on the importance of PA, summarizes the dimensions of PA, provides a standard operating procedure how to monitor PA using objective assessments and describes the psychometric properties of objectively measured PA. The present international task force recommends implementation of the standard operating procedure for PA data collection and reporting in the future. This should allow to further clarify the relationship between PA and clinical outcomes, to test the impact of treatment interventions on PA in individuals with COPD and to successfully propose a PA endpoint for regulatory qualification in the future.
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http://dx.doi.org/10.15326/jcopdf.2021.0213DOI Listing
August 2021

Objectively Measured Physical Activity as a COPD Clinical Trial Outcome.

Chest 2021 Jul 1. Epub 2021 Jul 1.

Department of Research and Development, CIRO, Horn, The Netherlands.

Background: Reduced physical activity is common in COPD and is associated with poor outcomes. Physical activity is therefore a worthy target for intervention in clinical trials; however, trials evaluating physical activity have used heterogeneous methods.

Research Question: What is the available evidence on the efficacy and/or effectiveness of various interventions to enhance objectively measured physical activity in patients with COPD, taking into account the minimal preferred methodologic quality of physical activity assessment?

Study Design And Methods: In this narrative review, the COPD Biomarker Qualification Consortium (CBQC) task force searched three scientific databases for articles that reported the effect of an intervention on objectively measured physical activity in COPD. Based on scientific literature and expert consensus, only studies with ≥ 7 measurement days and ≥ 4 valid days of ≥ 8 h of monitoring were included in the primary analysis.

Results: Thirty-seven of 110 (34%) identified studies fulfilled the criteria, investigating the efficacy and/or effectiveness of physical activity behavior change programs (n = 7), mobile or electronic-health interventions (n = 9), rehabilitative exercise (n = 9), bronchodilation (n = 6), lung volume reduction procedures (n = 3), and other interventions (n = 3). Results are generally variable, reflecting the large differences in study characteristics and outcomes. Few studies show an increase beyond the proposed minimal important change of 600 to 1100 daily steps, indicating that enhancing physical activity levels is a challenge.

Interpretation: Only one-third of clinical trials measuring objective physical activity in people with COPD fulfilled the preset criteria regarding physical activity assessment. Studies showed variable effects on physical activity even when investigating similar interventions.
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http://dx.doi.org/10.1016/j.chest.2021.06.044DOI Listing
July 2021

Validity and responsiveness of the Daily- and Clinical visit-PROactive Physical Activity in COPD (D-PPAC and C-PPAC) instruments.

Thorax 2021 03 21;76(3):228-238. Epub 2021 Jan 21.

Department of Respiratory Diseases, University Hospital Leuven, Leuven, Belgium.

Background: The Daily-PROactive and Clinical visit-PROactive Physical Activity (D-PPAC and C-PPAC) instruments in chronic obstructive pulmonary disease (COPD) combines questionnaire with activity monitor data to measure patients' experience of physical activity. Their amount, difficulty and total scores range from 0 (worst) to 100 (best) but require further psychometric evaluation.

Objective: To test reliability, validity and responsiveness, and to define minimal important difference (MID), of the D-PPAC and C-PPAC instruments, in a large population of patients with stable COPD from diverse severities, settings and countries.

Methods: We used data from seven randomised controlled trials to evaluate D-PPAC and C-PPAC internal consistency and construct validity by sex, age groups, COPD severity, country and language as well as responsiveness to interventions, ability to detect change and MID.

Results: We included 1324 patients (mean (SD) age 66 (8) years, forced expiratory volume in 1 s 55 (17)% predicted). Scores covered almost the full range from 0 to 100, showed strong internal consistency after stratification and correlated as a priori hypothesised with dyspnoea, health-related quality of life and exercise capacity. Difficulty scores improved after pharmacological treatment and pulmonary rehabilitation, while amount scores improved after behavioural physical activity interventions. All scores were responsive to changes in self-reported physical activity experience (both worsening and improvement) and to the occurrence of COPD exacerbations during follow-up. The MID was estimated to 6 for amount and difficulty scores and 4 for total score.

Conclusions: The D-PPAC and C-PPAC instruments are reliable and valid across diverse COPD populations and responsive to pharmacological and non-pharmacological interventions and changes in clinically relevant variables.
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http://dx.doi.org/10.1136/thoraxjnl-2020-214554DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7892393PMC
March 2021

Optimizing Pediatric Medicine Developments in the European Union Through Pragmatic Approaches.

Clin Pharmacol Ther 2021 Oct 2;110(4):871-879. Epub 2021 Feb 2.

Sanofi R&D, Chilly-Mazarin, France.

The European Union's Pediatric Regulation has strengthened the development of medicines for children in Europe through its system of obligations and rewards. However, opportunities remain to further optimize pediatric medicine developments, notably in relation to the implementation of the regulatory framework. This paper therefore describes bottlenecks identified by industry that occur during the medicinal development process, including those relating to the scientific advice process, pediatric investigation plan (PIP) development, compliance checks, and study submissions, and offers some considerations and insights to address these. Considerations, which are workable within the current legislative framework, focus on an integrated scientific discussion, optimization of PIP procedures and compliance checks, and an alignment of study-reporting requirements.
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http://dx.doi.org/10.1002/cpt.2152DOI Listing
October 2021

Correction to: How is the Pharmaceutical Industry Structured to Optimize Pediatric Drug Development? Existing Pediatric Structure Models and Proposed Recommendations for Structural Enhancement.

Ther Innov Regul Sci 2020 Sep;54(5):1085

Certara, Princeton, NJ, USA.

The article How is the Pharmaceutical Industry Structured to Optimize Pediatric Drug Development? Existing Pediatric Structure Models and Proposed Recommendations for Structural Enhancement, written by Thomas Severin et al. was originally published electronically on the publisher's internet portal on February 6, 2020 without open access. With the author(s)' decision to opt for Open Choice the copyright of the article changed on April 22, 2020 to © The Author(s) 2020 and the article is forthwith distributed under a Creative Commons Attribution 4.0 International License https://creativecommons.org/licenses/by/4.0/), which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.
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http://dx.doi.org/10.1007/s43441-020-00152-0DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7645507PMC
September 2020

Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines.

Br J Clin Pharmacol 2020 06 24;86(6):1034-1051. Epub 2020 Apr 24.

European Union Network for Health technology assessment (EUnetHTA), The Netherlands.

The understanding of the benefit risk profile, and relative effectiveness of a new medicinal product, are initially established in a circumscribed patient population through clinical trials. There may be uncertainties associated with the new medicinal product that cannot be, or do not need to be resolved before launch. Postlicensing or postlaunch evidence generation (PLEG) is a term for evidence generated after the licensure or launch of a medicinal product to address these remaining uncertainties. PLEG is thus part of the continuum of evidence development for a medicinal product, complementing earlier evidence, facilitating further elucidation of a product's benefit/risk profile, value proposition, and/or exploring broader aspects of disease management and provision of healthcare. PLEG plays a role in regulatory decision making, not only in the European Union but also in other jurisdictions including the USA and Japan. PLEG is also relevant for downstream decision-making by health technology assessment bodies and payers. PLEG comprises studies of different designs, based on data collected in observational or experimental settings. Experience to date in the European Union has indicated a need for improvements in PLEG. Improvements in design and research efficiency of PLEG could be addressed through more systematic pursuance of Scientific Advice on PLEG with single or multiple decision makers. To date, limited information has been available on the rationale, process or timing for seeking PLEG advice from regulators or health technology assessment bodies. This article sets out to address these issues and to encourage further uptake of PLEG advice.
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http://dx.doi.org/10.1111/bcp.14279DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7256124PMC
June 2020

Shared Learnings on the New EMA First-in-Human and Early Clinical Trial Guideline: Proceedings From a DIAlogue Session at DIA Europe 2018.

Ther Innov Regul Sci 2020 03 6;54(2):462-467. Epub 2020 Jan 6.

Preclinical Safety, Novartis Pharma, Basel, Switzerland.

The EU is a member of the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH), and therefore adopts the ICH Guidelines, including the ICH M3 Guideline on Nonclinical Safety Studies. Following the 2016 incident in France with BIA 10-2474, and in light of the substantial evolvement of how early clinical development has been undertaken during the last 10 years, for example, conducting integrated (FIH) studies that include multiple parts (eg, single ascending doses, multiple ascending doses, food effect), EMA decided to update the existing 2007 FIH guideline. The key revisions to the 2007 guideline, now titled "Guideline on Strategies to Identify and Mitigate Risks for First-in-Human and Early Clinical Trials With Investigational Medicinal Products," include additional information. The revision reinforces the importance and impact of pharmacologic data, which supports the intended efficacy of the compound, risk assessment, and protocol design. The updates, effective February 2018, are intended to provide additional guidance and clarity for Sponsors developing FIH and early phase clinical research programs, and ultimately support subject safety. At the 2018 DIA Europe Annual Meeting in Basel, Switzerland, European regulators, industry representatives and academics convened a DIAlogue Session on April 17 to discuss how the revised 2017 guideline is being applied, and to establish recommendations for its application. Using two case studies as examples, the session participants discussed the nonclinical and clinical considerations for applying the newly revised recommendations, and interacted with a panel including regulators and industry representatives. The proceedings from this session reflect practical considerations for the implementation of the revised guideline.
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http://dx.doi.org/10.1007/s43441-019-00077-3DOI Listing
March 2020

How is the Pharmaceutical Industry Structured to Optimize Pediatric Drug Development? Existing Pediatric Structure Models and Proposed Recommendations for Structural Enhancement.

Ther Innov Regul Sci 2020 09 6;54(5):1076-1084. Epub 2020 Feb 6.

Certara, Princeton, NJ, USA.

Background: Pediatric regulations enacted in both Europe and the USA have disrupted the pharmaceutical industry, challenging business and drug development processes, and organizational structures. Over the last decade, with science and innovation evolving, industry has moved from a reactive to a proactive mode, investing in building appropriate structures and capabilities as part of their business strategy to better tackle the challenges and opportunities of pediatric drug development.

Methods: The EFGCP Children's Medicines Working Party and the IQ Pediatric working group have joined their efforts to survey their member company representatives to understand how pharmaceutical companies are organized to fulfill their regulatory obligations and optimize their pediatric drug development programs.

Results: Key success factors and recommendations for a fit-for-purpose Pediatric Expert Group (PEG) were identified.

Conclusion: Pediatric structures and expert groups were shown to be important to support optimization of the development of pediatric medicines.
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http://dx.doi.org/10.1007/s43441-020-00116-4DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7458895PMC
September 2020

Orphan Medicines for Pediatric Use: A Focus on the European Union.

Clin Ther 2019 12 5;41(12):2630-2642. Epub 2019 Nov 5.

Sanofi R&D, Chilly-Mazarin, France. Electronic address:

Purpose: European policy makers have provided a number of incentives for the development of medicines for orphan diseases as early as 1999 through the Orphan Regulation and created obligations for medicines developers to investigate their products in children through the Paediatric Regulation adopted in 2006. This article describes the challenges that developers of orphan medicines are facing with pediatric indications, discusses the interplay between the Orphan Regulation and the Paediatric Regulation, and provides some recommendations on how to optimize drug development under the current European Union regulatory framework.

Methods: This article discusses the European Union's Orphan Regulation, Paediatric Regulation, and the implications of the intersection of the regulations on the development of orphan medicines for pediatric use.

Findings: Although these regulations have been successful in meeting their objectives separately, different regulatory frameworks entail separate governance, multiple assessments, varying approaches and priorities to unmet medical needs, and joined-up regulatory process coordination. Better integration of regulatory pathways would therefore be helpful in stimulating more global drug development of pediatric orphan medicines, including optimizing the interaction between both regulations, using innovative drug development approaches while considering alternatives to randomized clinical trials, better identification and prioritization of unmet medical needs in pediatrics, and ensuring the alignment of regulatory processes.

Implications: Rare diseases are categorized as "orphan diseases" because their occurrence in a small number of patients means that, regardless of the apparent high unmet medical need, there is limited public and market interest to justify the high development risk and significant investment to develop new treatments. However, unexplored potential within the area, as well as a conducive regulatory environment, can further support the development of medicines to treat rare diseases, including for children.
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http://dx.doi.org/10.1016/j.clinthera.2019.10.006DOI Listing
December 2019

Industry Perspective on Using MIDD for Pediatric Studies Requiring Integration of Ontogeny.

J Clin Pharmacol 2019 09;59 Suppl 1:S112-S119

Certara Strategic Consulting, Princeton, NJ, USA.

Joining the Food and Drug Administration/University of Maryland Center of Excellence in Regulatory Science and Innovation Workshop to discuss and identify solutions to optimize pediatric drug development and, in particular, to address the question as to whether we are ready to incorporate pediatric ontogeny into modeling was the opportunity to share learnings, confront ideas, and present examples of studies performed in industry and academia. This was not only the opportunity to reflect on the experience and the knowledge so far within the current regulatory framework but also to look at the future and explore new and future approaches as well as best practices with the use of modeling and simulation and extrapolation as part of pediatric development.
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http://dx.doi.org/10.1002/jcph.1495DOI Listing
September 2019

Recognizing that Evidence is Made, not Born.

Clin Pharmacol Ther 2019 04 4;105(4):844-856. Epub 2019 Jan 4.

Canadian Arthritis Patient Alliance, Midland, New Brunswick, Canada.

Therapeutic product development, licensing and reimbursement may seem a well-oiled machine, but continuing high attrition rates, regulatory refusals, and patients' access issues suggest otherwise; despite serious efforts, gaps persist between stakeholders' stated evidence requirements and actual evidence supplied. Evidentiary deficiencies and/or human tendencies resulting in avoidable inefficiencies might be further reduced with fresh institutional cultures/mindsets, combined with a context-adaptable practices framework that integrates emerging innovations. Here, Structured Evidence Planning, Production, and Evaluation (SEPPE) posits that evidence be treated as something produced, much like other manufactured goods, for which "built-in quality" (i.e., "people" and "process") approaches have been successfully implemented globally. Incorporating proactive, iterative feedback-and-adjust loops involving key decision-makers at critical points could curtail avoidable evidence quality and decision hazards-pulling needed therapeutic products with high quality evidence of beneficial performance through to approvals. Critical for success, however, is dedicated, long-term commitment to systemic transformation.
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http://dx.doi.org/10.1002/cpt.1317DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6590384PMC
April 2019

Medicines Adaptive Pathways to Patients: Why, When, and How to Engage?

Clin Pharmacol Ther 2019 05 11;105(5):1148-1155. Epub 2018 Sep 11.

Astra Zeneca, Paris, France.

Medicines Adaptive Pathways to Patients (MAPPs) seeks to foster access to novel beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span, in a sustainable fashion. We summarize the MAPPs engagement process and critical questions to be asked at each milestone of the product life-span. These considerations are of relevance for regulatory and access pathways that strive to address the "evidence vs. access" conundrum.
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http://dx.doi.org/10.1002/cpt.1121DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6585618PMC
May 2019

Challenges and Opportunities in the Development of Medical Therapies for Pediatric Populations and the Role of Extrapolation.

Clin Pharmacol Ther 2018 03 31;103(3):419-433. Epub 2018 Jan 31.

Global Clinical Development, Bayer HealthCare, Basel, Switzerland.

Extrapolation can be used to address challenges in pediatric drug development. This review describes how these challenges could be addressed by further evolution of quantitative frameworks (i.e., model-based/informed drug discovery and development) and regulatory science in support of pediatric drug development. Included are examples of diseases/indications where extrapolation has been used in different ways as a basis for identifying gaps in the framework and opportunities for continued advancement of pediatric drug development.
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http://dx.doi.org/10.1002/cpt.1000DOI Listing
March 2018
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